4. Special cases in marketing authorisations: Advanced therapy medicines (ATMPs)

In this regulatory framework ATMPs are defined and classified into the following types according to criteria established in the relevant legislation:

Figure 3

The regulatory framework for ATMPs further encompasses the following elements (adapted from the EMA):

• the centralised marketing authorisation procedure (see lesson 1 this course) which is obligatory for ATMPs. However, according to recital 7, the ATMP Regulation does not affect decisions of individual Member States about the use of embryonic stem cells or animal cells which may still be covered by national legislation.
• the CAT: an EMA multidisciplinary expert Committee (Committee for Advanced Therapies).
  Main task: to assess advanced therapy products and prepare a draft opinion before the CHMP adopts a final opinion and the authorisation is granted by the Commission
  Other tasks with participation of the CAT:
  • classification of ATMPs: The CAT delivers scientific recommendations on ATMP classification after consultation with the European Commission.
  • certifying quality and non-clinical data for small and medium-sized enterprises (SMEs) developing ATMPs;
  • contributionto scientific advice, in cooperation with the Scientific Advice Working Party (SAWP);
  • delivery of  advice on the conduct of efficacy follow-up, pharmacovigilance or risk-management systems for ATMPs;
  • advice to the CHMP on any medicine that may require expertise in ATMPs for the evaluation of its quality, safety or efficacy;
  • scientific assistance  in developing any documents relating to the objectives of the Regulation on ATMPs;
  • provision of scientific expertise and advice for any Community initiative related to the development of innovative medicines and therapies requiring expertise on ATMPs;
  • support to  the work programmes of the CHMP working parties.
• technical requirements adapted to the particular characteristics of these products